Higher dose of Spinraza improves motor function in infants with spinal muscular atrophy

Topline data from an ongoing clinical trial of a higher dose regimen of Spinraza in treatment-naive, symptomatic infants with spinal muscular atrophy showed significantly improved motor function at 6 months.

According to a press release from drug manufacturer Biogen, the phase 2/3 DEVOTE study evaluated the safety and efficacy of two 50 mg doses of antisense oligonucleotide Spinraza (nusinersen) given 14 days apart, along with a higher maintenance regimen of 28 mg every 4 months, compared with the approved nusinersen regimen.

Data showed the primary endpoint of statistically significant improvement in motor function was attained in those who received the higher dose regimen compared with a prespecified matched sham group from the phase 3 ENDEAR clinical trial.

“Development of the higher dose nusinersen regimen is one of Biogen's core approaches to address the unmet need that exists in [spinal muscular atrophy] and seek better outcomes for patients,” Stephanie Fradette, PharmD, head of the neuromuscular unit at Biogen, told Healio in an email. “We are encouraged by the results and look forward to sharing additional details with the community.”

Researchers measured improvements based on changes from baseline on the Children's Hospital of Philadelphia-Infant Test of Neuromuscular Disorders (CHOP-INTEND) scale at 6 months, comparing the higher dose regimen of nusinersen with the ENDEAR sham cohort.

DEVOTE is a three-part study that enrolled 145 patients across ages and SMA types. The current analysis included an active control, randomized treatment cohort comprised of 75 young children with infantile-onset SMA. They were randomly assigned 2:1 to receive the investigational higher dose regimen of nusinersen or an approved 12 mg regimen of four loading doses as well as maintenance doses every 4 months.